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Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular Dystrophy

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PTC Therapeutics

Status and phase

Completed
Phase 2

Conditions

Duchenne Muscular Dystrophy

Treatments

Drug: Ataluren

Study type

Interventional

Funder types

Industry

Identifiers

NCT02819557
PTC124-GD-030-DMD

Details and patient eligibility

About

This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to <5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.

Full description

In nonsense mutation DMD (nmDMD), early start of treatment is important and necessary and, therefore, it is relevant to understand the correct and tolerable dose in this age group, particularly since ataluren is dosed by weight. This study included a 4-week screening period, a 52-week treatment period (the first 4 weeks of which included PK parameters), and a 4-week follow-up period for participants who completed the treatment period (60 weeks total duration). The objective of the extension period (treatment period after PK parameters have been completed) was to assess the long-term safety of chronic administration of ataluren in this participant population.

Enrollment

14 patients

Sex

Male

Ages

2 to 5 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

  • Males ≥2 to <5 years of age
  • Body weight ≥12 kg
  • Diagnosis of DMD
  • Nonsense mutation in at least 1 allele of the dystrophin gene

Exclusion criteria

  • Participation in any other drug or device clinical investigation
  • Ongoing use of prohibited concomitant medications

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

14 participants in 1 patient group

Ataluren
Experimental group
Description:
Participants will be administered ataluren orally at a dose of 10 milligrams/kilograms (mg/kg) in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening (for a total of 40 mg/kg/day) for up to 52 weeks. Dose will be provided based upon the weight of each participant, which will be assessed every 12 weeks.
Treatment:
Drug: Ataluren

Trial documents
2

Trial contacts and locations

6

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Data sourced from clinicaltrials.gov

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